Full Press Release Details
Press Release, Dated July 31, 2017
Geron Announces Updates to Imetelstat
Clinical Development
Conference Call Scheduled for 8:00
a.m. ET on Tuesday, August 1
MENLO PARK, Calif., July 31, 2017
-- Geron Corporation (Nasdaq: GERN) today
announced updates to the clinical development plans for IMerge and IMbark, the
ongoing trials of the telomerase inhibitor imetelstat in lower risk
myelodysplastic syndromes (MDS) and relapsed or refractory myelofibrosis (MF),
respectively, being conducted by Janssen Research & Development, LLC. For
IMerge, Part 1 will be expanded to enroll additional patients in a refined MDS
population to confirm the clinical benefit and safety observed from current
results. For IMbark, the trial remains unchanged. Geron expects that the IMbark
protocol-specified primary analysis, the completion of which triggers a future
Continuation Decision by Janssen, will begin no later than the third quarter of
Original Trial Design
IMerge (NCT02598661) is a Phase 2/3
clinical trial evaluating imetelstat in transfusion dependent patients with Low
or Intermediate-1 risk MDS who have relapsed after or are refractory to prior
treatment with an erythropoiesis stimulating agent (ESA). The clinical trial is
in two parts: Part 1 is a Phase 2, open-label, single-arm design in
approximately 30 patients and Part 2 is designed to be a Phase 3, randomized,
controlled trial in approximately 170 patients. The primary efficacy endpoint is
the rate of red blood cell (RBC) transfusion independence (TI) lasting at least
In Part 1 of IMerge, 32 patients were
enrolled, of which a subset of 13 patients had not received prior treatment with
either a hypomethylating agent (HMA) or lenalidomide and did not have a del(5q)
chromosomal abnormality. As of May 2017, the 13-patient subset showed an
increased durability and rate of transfusion independence compared to the
overall trial population ( 8-week RBC-TI: 53.8% vs 34.4%). The safety profile
in Part 1 was consistent with prior clinical trials of imetelstat in hematologic
malignancies, and no new safety signals were identified. The most common adverse
events were cytopenias, which were manageable, and included grade 3/4
neutropenia and thrombocytopenia.
Based on these data from the 13-patient
subset, the Joint Steering Committee has decided to amend Part 1 of the protocol
to enroll approximately 20 additional patients who are non-del5q and na ve to
HMA and lenalidomide treatment in order to increase the experience and confirm
the benefit-risk profile of imetelstat dosed at 7.5 mg/kg every four weeks in
this refined target patient population. Enrollment into the expanded Part 1 is
expected to begin in the fourth quarter of 2017.
Separately, a data package and proposed
refinements to the trial design for Part 2 of IMerge were previously provided to
the FDA following an internal data review completed by Janssen in April, and
related interactions are ongoing. Feedback from ongoing FDA interactions, data
from the expanded Part 1, and other imetelstat program information, including
the protocol-specified primary analysis for IMbark, are expected to inform
Janssen s decision of whether to move forward to Part 2 of IMerge.
Detailed results for the original 32 patients in Part 1 of IMerge, including key secondary endpoints of hematologic improvement and rate
of RBC-TI lasting at least 24 weeks, as well as duration of response and
detailed safety information, will be submitted for presentation at a major
IMbark (NCT02426086) is a Phase 2 trial in
patients with Intermediate-2 or High Risk MF who have relapsed after or are
refractory to prior treatment with a JAK inhibitor. The trial continues without
modification, and patients remaining in the treatment phase may continue to
receive imetelstat. All safety and efficacy assessments will be conducted as
planned in the protocol, which includes an assessment of a potential survival
benefit associated with imetelstat treatment. To date, median overall survival
has not yet been reached in either the 4.7 mg/kg
or 9.4 mg/kg dosing arm. Enrollment of new patients to the trial remains
suspended because the total number of patients enrolled to date is adequate to
perform the protocol-specified primary analysis. Geron expects Janssen to
perform an internal data review in the first quarter of 2018 to enable a
potential protocol amendment to allow the long-term treatment and follow-up of
patients, including for survival, beyond the current April 2018 per-protocol
Continuation Decision
The Joint Steering Committee has agreed
that the timing of the protocol-specified primary analysis for IMbark will begin
upon the earlier of either a pre-specified number of deaths occurring in the
trial or the end of the third quarter of 2018. Following completion of this
primary analysis, which includes an assessment of potential survival benefit
associated with imetelstat treatment, Janssen will notify Geron whether it
elects to maintain the license rights and continue the development of imetelstat
in any indication, i.e., the Continuation Decision.
At 8:00 a.m. EDT on August 1, 2017,
Geron s management will host a conference call to discuss these updates to
imetelstat clinical development. Participants can access the conference call
live via telephone by dialing 877-303-9139 (U.S.); +1-760-536-5195
(international). The conference ID number is 42220500. A live audio-only webcast
is also available through the company s website at www.geron.com in the
Investors section under Events and at http://edge.media-server.com/m/p/oqg6hyn5.
The audio webcast of the conference call will be available for replay
approximately one hour following the live broadcast through September 1,
Imetelstat (GRN163L; JNJ-63935937) is a
potent and specific inhibitor of telomerase that is administered by intravenous
infusion. This first-in-class compound, discovered by Geron, is a specially
designed and modified short oligonucleotide, which targets and binds directly
with high affinity to the active site of telomerase. Preliminary clinical data
suggest imetelstat has disease-modifying activity by inhibiting the progenitor
cells of the malignant clones associated with hematologic malignancies in a
relatively select manner. Most commonly reported adverse events in imetelstat
clinical studies include fatigue, gastrointestinal symptoms and cytopenias.
Imetelstat has not been approved for marketing by any regulatory authority.
About the Collaboration with Janssen
On November 13, 2014, Geron entered into
an exclusive worldwide license and collaboration agreement with Janssen Biotech,
Inc., to develop and commercialize imetelstat for oncology, including
hematologic myeloid malignancies, and all other human therapeutics uses. Under
the terms of the agreement, Geron received an upfront payment of $35 million and